Neurological Effects and Safety of Encapsulated Dexamethasone Sodium Phosphate in Pediatric Ataxia Telangiectasia: Insights from the NEAT Phase 3 Trial

Neurological Effects and Safety of Encapsulated Dexamethasone Sodium Phosphate in Pediatric Ataxia Telangiectasia: Insights from the NEAT Phase 3 Trial

The NEAT phase 3 trial evaluated erythrocyte-encapsulated dexamethasone sodium phosphate in children with ataxia telangiectasia aged 6–9 years, confirming safety but failing to demonstrate statistically significant neurological improvement over placebo.
Evaluating Cell-Free DNA Testing for Autosomal Recessive Conditions in General-Risk Pregnancies: A Multicenter Prospective Study

Evaluating Cell-Free DNA Testing for Autosomal Recessive Conditions in General-Risk Pregnancies: A Multicenter Prospective Study

This prospective multicenter study demonstrates that cfDNA fetal risk screening accurately identifies autosomal recessive conditions in general-risk pregnancies with high specificity and improved positive predictive value compared to traditional carrier screening, supporting its potential as a primary screening tool.